Tag Archives: saatchibill

Telegraph: ‘This isn’t quackery, it’s good practice’

The Bill Team’s Dominic Nutt talks to Prof Walker, University of Nottingham

Paediatric cancer specialist Prof David Walker’s work is a bittersweet affair. At times he is able to deliver the happiest of news to parents: that he and his team have saved the life of their child. Equally, he is the one who has to tell a fearful mother and father that there’s nothing more he can do, that their son or daughter’s cancer has spread beyond the reach of his drugs and the surgeon’s knife, and that their child will die.

Based at Nottingham University for the past 22 years, working at Nottingham Children’s Hospital and Nottingham Medical School, Prof Walker combines a passionate commitment to the welfare of his patients with a deep respect for scientific discipline.

He wants to see more and better therapies added to the armoury of paediatric cancer treatments, to save the lives of more children, and is frustrated by the obstacles that block the path to new cures for cancers.

Medical science decrees that no new treatment should be used until it has been thoroughly tested in randomised clinical trials. Trials are the gold standard, Prof Walker stresses, and where possible patients should be in a trial if one is available. “Research does require regulation to introduce new drugs and treatment safely,” he says.

But trials alone cannot meet the desperate need for new treatments, in particular for rare cancers. “There will never be enough trials, they take years, and in any event there are rarely trials for less common diseases. There just aren’t enough patients.”

Around one in 600 children under 16 are diagnosed with cancers. The most common, such as leukaemia, account for a third of cases, and treatment success rates are high. But some are hard to treat, other than with surgery, such as the highly malignant rhabdoid tumours, which start in the kidney. There are no standard therapies for these cancers and less than one in five children diagnosed with a stage III or IV rhabdoid tumour will survive beyond four years.

With such rare childhood cancers, it is hard to gather enough patients to form a trial in one country alone, and these have to be organised internationally.

“The problem is that there are many different tumour types and sub-types in childhood cancers, each having an unique biological signature justifying a unique scientific rationale for their treatment with modern drugs in development,” says Prof Walker. “They are all rare in the population and there is a huge element of luck as to whether in your child’s case there is a trial available.”

So, while potential new drugs may exist, they cannot be used. “A drug may be available, the scientific rationale for its use may exist, but patients cannot receive it if no trial has been organised to assess its effectiveness and toxicity,” says Prof Walker.

He also points out that the “big four” cancers – breast, bowel, lung and prostate – dominate media coverage as well as fundraising, research and trials. Researchers want to work on the common cancers because their research will be better funded, while drugs companies are more interested in this area as there is a bigger market, and a greater potential profit.

If you have a less common cancer – and there are hundreds – your chances of getting on a trial are limited and the chances of there being innovative treatments are, in many cases, zero. Yet taken together, less common cancers – defined as a cancer that affects five people or fewer in 10,000 – account for more than half of all British cancer deaths. One in six of us will develop and die from a rare cancer.

The lack of trials in this area means doctors who want to cure their patients, rather than just manage their deaths, are caught in a scientific circular argument. There is no evidence that a new treatment will work, so it cannot be used to find out if it does work.

Prof Walker believes that doctors should be able to try new treatments with such patients on an individual basis. Yet, he argues, the law, and the culture of defensive medicine which surrounds it, stands in the way of innovation. Doctors are protected if they stick to the well-worn path of “standard procedure” even if it leads to the death of the patient. But they may be vulnerable to legal action if they try something new and it fails.

Which is why Prof Walker supports Maurice Saatchi’s Medical Innovation Bill, currently going through a public consultation process by the Department of Health, which ends later this month. It seeks to provide legal protection for doctors who innovate in the interests of their patients. He argues that when patients are terminally ill and there is no trial for which they are eligible, a doctor should be free to innovate.

“We need to allow them to try new drugs outside of a formal trial and collect the data from those innovations to inform the next generation of trials,” he says. “The Saatchi Bill would protect individual doctors who try new, untrialled treatments, where there is a scientific rationale for their use, in patients who consent.

“This isn’t a licence for the maverick doctor acting alone – the Bill obliges the doctor to seek agreement from peers.”

The Saatchi Bill, he believes “will give the patients and their families additional choice and allow doctors to try new medicines in people who have nowhere else to go, and do it in such a way as we could all learn from it”.

Prof Walker also believes that even when an individual patient cannot be cured, this kind of innovation will advance medical science for future patients.

“When people are dying,” he says, “they all would like their passage through their illness to have some meaning and to learn from the loss of their life.”

He also explains that many cancer drug trials rightly focus hard on a tightly defined group of patients with the same tumour type, which has within it a particular cancer molecule, that the drug being tested is designed to block.

Using molecularly targeted drugs in patients with the same tumour drives robust results. But, says Prof Walker, “there is a weakness in the process, because it doesn’t tell you where else the new drug may work. It only tells you that the drug works on a very specific patient type with a very specific tumour which has within it that particular molecular target.”

The new drug may work in another rarer cancer with the same molecular target, he says. “But if I have a patient with a very rare cancer with the same molecular target, I can’t use it. Current rules require us to set up another trial in that tumour, and that’s expensive and requires collaboration with the drug industry, which may not wish to supply the drug for such a purpose if they don’t think it is commercially advantageous. So, in the meantime, the hospital won’t release that licensed drug for an unlicensed purpose because there’s no evidence that it works,” he says.

This is where the Lord Saatchi’s Medical Innovation Bill would help, he argues.

“If we could test drugs in a patient and record our results in an open-access database, this would contribute to our understanding of the application of this drug and perhaps help with selecting new drugs for clinical trial by identifying those with most promise; although it is important that the drug has already been tested fully on patients with a different cancer but with the same molecular signature.”

Opponents of the Bill argue that a law that supports doctors who want to try new treatments outside the trial process is a “quack’s charter”.

Prof Walker disagrees: “To offer an untried treatment and not learn from the process would be quackery.” “But to use untried treatment and to try to learn, responsibly, about the effect on the individual, and share that learning with others would seem to be not quackery, but actually responsible professional practice.”

Sign the petition for the Medical Innovation Bill: http://chn.ge/1pqY6lS

 

This Bill is very important, as the scientific world has changed

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By Charlie Chan, Consultant General Surgeon, Nuffield Health

In spite of medical advances over the last 50 years, many people across the world still die prematurely from cancer, heart disease, lung disease, and terrible degenerative diseases like muscular dystrophy and motor neurone disease.

For many of these people, the options for treatment and run out quickly, with no prospect of improvement in quality of life or life expectancy. For some, but not all, exploring new treatment in a responsible manner is something that the individuals may wish to do, if only to benefit future generations.

This Bill is very important, as the scientific world has changed. The last generation has seen an enormous change in our fundamental understanding of diseases and their processes.

Sign the petition for the Medical Innovation Bill: http://chn.ge/1pqY6lS

This has been underpinned by the massive changes in laboratory research, genetics, 3-D printing, and regenerative medicine. This knowledge could not have been foreseen over 50 years ago, when the Bolam case was first heard.

This new knowledge has been underpinned by the vast improvement in computer technology, which has benefited all parts of society. When treating patients and diseases, doctors and scientists are waging a conflict on 2 fronts.

The frontline remains the one of the individual patient, with the doctor at the bedside or in the operating theatre. Behind-the-scenes, there is a continuing intelligence campaign underpinned by scientific research in the laboratory.

100 years ago, communication between the front and Intelligence Corps might well have been done by carrier pigeon. Now, information exchange occurs rapidly in real-time between GCHQ and troops in Afghanistan. In medicine however, this communication or translation of new scientific knowledge to the bedside remains slow.

We are fast approaching and era when scientific research may outstrip our ability to deliver this to the patients. Hence, we need to address a new way to deliver innovative treatments.

That is not to say that the standard clinical trial model is dead. There is still much merit in the randomised trial as a paradigm. It is vitally important that the development of new standard treatments for large numbers of people is underpinned by solid statistical analysis and estimation of perceived benefits.

However, a new process for innovative treatments may provide many new hypotheses for new trials, which can then cement new techniques and drugs.

There are some colleagues who rightly have concerns that a change in the law may constitute a charlatan’s charter. However, the Bill contains safeguards to ensure that there is no quackery. All standard and trial treatments need to be exhausted, there needs to be logic behind the proposed treatment, and this treatment needs to be agreed within the peer group, prior to discussion with the patient.

The agreement within the peer group will need to be done in a timely manner. There is a significant challenge for the profession to establish a framework for such peer group discussions, which may occur on the local level or through a National Specialty Association.

However, it is well recognised that this must be something that can be done quickly for patients treated in a district general hospital by local consultants, as well as those managed in large teaching hospitals.

Some of my colleagues would strongly support some form of central data collection.

A central data repository to be analysed on a regular basis, in order to establish whether any putative innovative treatments have any merit for further investigation in large clinical trials.

This might be located in an academic university department, so that this might be independent of central government control.

The future is extremely exciting. Basic scientific research has enhanced greatly our fundamental understanding of many diseases, such as cancer.

This understanding of the basic diseases means that some new biological drugs may have multiple applications across different cancer types. It makes logical sense to exploit this basic science knowledge, particularly to benefit those patients with rare diseases, for whom a standard clinical trial may be impractical or financially non-viable.

Advances in regenerative medicine, particularly in the USA, now mean that organs can be printed in a matter of hours or weeks.

This will herald a completely different way in which we might manage people with cardiac, kidney, or liver disease.

We must grasp this opportunity to change things for future generations, otherwise these scientific advances may be for naught.

About the author:

Charlie Chan is a Consultant General Surgeon with a special interest in breast disease, skin cancer, and soft tissue tumours. He also has a varied practice in general surgery. He has extensive research interests, and is currently involved on the Trial Management Group for 4 large UK breast cancer trials.

On average, he performs 30 to 40 major breast reconstructions a year, as well as numerous cosmetic breast procedures. Amongst his breast cancer patients, he is normally able to conserve the breast in 70-75% of his patients. AIong with his colleagues in Cheltenham, James Bristol and Fiona Court, he is one of only a few surgeons in the UK who are trained in the new Breform™ cosmetic breast operation. He has contributed to numerous articles in the national press (Sunday Telegraph, Daily Telegraph, Daily Mail) as well as the local press and BBC Radio Gloucestershire.

He has written breast cancer guidelines for the Association of Breast Surgeons, organised national cancer surgery meetings for the British Association Of Surgical Oncology, and reviews oncology education across Europe for the Accreditation Council of Oncology in Europe.

Sign the petition for the Medical Innovation Bill: http://chn.ge/1pqY6lS

Cardiff consultation: Answers to questions that came via Twitter

During the recent Department of Health public consultation meeting in Cardiff (18 March 2014) attended by doctors, academics and patients, we got some questions on Twitter we discussed.

Sean Kielthy – @LMKPartnership – in particular asked three questions which sum up what many others are asking.

Sean asked:

1. ‘Defining experts [in the Bill]. A homeopath may be an expert in their field. How do we stop them getting in?’

Answer: The Bill can only be used by qualified and registered doctors. The term ‘doctor’ is a legal one and homeopaths (for example) would not be protected by the Bill, if it became law.

Basically, you have to be a legally-recognised doctor. If not, the Bill doesn’t cover you.

2. ‘How do we keep the quacks out? [I want] science-based medicine only’.

Answer: Apart from the answer above – that you must be a doctor to be covered by the Medical Innovation Bill – you must also get the agreement of a panel of medical experts and other doctors before you offer a new treatment to the patient.

It is inconceivable that a quack doctor, treating a terminally ill patient, who comes up with a crazy ‘snake-oil’ treatment would get the support of a panel of other senior doctors.

To get agreement, the doctor will have to produce evidence or coherent reasons and a theory as to why the new treatment is worth considering.

The supporting doctors will be named as part of the sign-off process. At the moment, it is possible for a doctor to act alone without the agreement of senior and qualified peers.

So the Bill makes it harder for a quack to prey on a patient. After the Bill is passed, they will not be able to act alone.

3. ‘People in these situations [patients who are very ill] can be desperate. [I] don’t want to see quackery used and funding taken from science.’

Answer: Quacks will not be able to hide behind the Bill. In fact the Bill will expose quacks.

There is another question here, though – the cost of innovation. Will the Bill cost money? There is no logical reason to say that it will. An innovation may cost, it may cost less or it may cost nothing.

For example, it may conceivably be an innovation to do nothing. There are examples where it is felt that adjuvant chemotherapy for certain cancers may be at best only marginally beneficial, and that the associated potential side-affects of the treatment may outweigh the standard chemo treatment.

In this case, doing nothing would be an innovation. (This is not to advocate in any way that not having chemo is a good thing – it is simply a generic example).

However, if people argue that innovation costs money and that the Bill will inspire innovation and new and better treatments, then that is not an argument against the Bill – it’s an argument against all medical innovation and medical progress.

The Bill won’t divert resources from science. The Bill will help individual doctors help individual patients on a one-off basis.

By trying new ideas and techniques in this way, doctors will learn and the data they collect can be used by other doctors and scientists to inspire full medical trials and scientific discovery.

The Medical Innovation Bill works hand-in-hand with science to deliver new treatments for hard-to-cure diseases.

 

Supporting innovation – exposing the maverick

The Saatchi Bill will make it much easier for doctors to innovate safely in the interests of their patients.

At the same time, it will expose the doctor who wishes to exploit their patient, preying on them and their vulnerability in order to attempt a reckless experiment.

Doctors will not be protected by the Saatchi Bill unless they go through a rigorous and specific process to ensure that the attempted innovation is the right course of action for the patient.

As the draft Bill states in paragraph 2 (3a) a doctor wishing to try a new treatment – for example in the case where standard treatments aren’t working – must consult a body of senior and relevant medical experts and get their consent.

The doctor must also record their opinion, including and dissenting voices.

The decision of the panel of experts must then be presented to the patient – including any contrary opinions if there are any – and the patient must of course also agree to go through with the innovative treatment.

Finally, the note of the opinions must then be attached to the patient’s consent form as a permanent record.

Only then will the doctor be legally covered by the Bill.

The Bill imposes a much higher standard of consent than other health legislation.

For example in order to section a patient, the Mental Health Act requires the signatures of only two doctors.

Currently, it is easier now for a doctor to indulge in reckless experimentation and maverick medicine, than it will be if the Bill becomes law.

As it now stands – without the Saatchi Bill – a doctor can attempt to persuade a vulnerable patient to embark on a dangerous treatment path.

This is so because the doctor technically doesn’t need to refer to a panel of experts before trying the non-standard procedure. He or she can act alone.

So, the Medical Innovation Bill supports and encourages reasoned innovation – and exposes the maverick.

Lord Woolf: Former Master of the Roles and Lord Chief Justice:

[The Bill] could give confidence to medical practitioners engaged in the field of treatment of cancer that in appropriate circumstances they could safely recommend and implement a course of treatment, or non-treatment , which some, or indeed the majority of their professional colleagues, might regard as unorthodox.

I have come to this conclusion because it is, in my view, undoubtedly the case that there is insufficient certainty as to the course which courts will adopt in this country at the present time when faced with an allegation that a medical practitioner’s treatment of a patient was inappropriate because of its innovative nature.

[There is] a risk that the present state of the law could inhibit the proper development of treatment of particular cancers.

Any way of avoiding this by legislation, in my opinion, should be welcomed. I would therefore hope that in the Lords, at any rate, your Bill will be well received.

About Lord Woolf

Consultation launches in the House of Lords


Last week cancer bloggers, tweeters and patients united to make medical history in the House of Lords – at a Google Hangout in support of Maurice Saatchi’s Medical Innovation Bill.

Together we launched the Saatchi Bill public consultation – backed by the Department of Health and Secretary of State @Jeremy_Hunt.

The Bill will allow patients to ask for – and doctors to deliver – new and innovative medical treatments for rare and hard-to-cure diseases without fear of being sued.

The Government backs our Bill – and promises to pass it into law, if the public consultation is successful.

It all hangs in the balance right now. Nothing is certain.

But if the response we got from those in the room who came to hear Maurice Saatchi, patients, families and professors speak up for the Bill is anything to go by, we have a fighting chance.

You joined us in a Google Hangout and heard the arguments of Debbie Binner who lost her daughter, Chloë to a rare bone cancer, to Chloë’s best friend Mike Thomas and to Mavis Nye who is dying of mesothelioma.

They all want the Bill. They want innovation – not the endless repetition of failed medical treatments.

After the Hangout, #SaatchiBill twitter and blog conversations went ballistic. We want more of that and we want hundreds of people to respond to the government’s public consultation so that no politician can say the Bill is not needed or wanted.

As Mavis Nye told the room: “I’m terminally ill. I shouldn’t have to chase treatment. My oncologist can only give me what’s laid down by the national health system. I back this innovation Bill. I want it and I want it today – because I might not be here tomorrow.”

 

In Memory of Chloë Drury

Chloe Drury by Mike Thomas

I gripped the wooden lectern tightly with both hands to try and prevent myself breaking down with emotion.

It was a year ago last Friday. The lectern was at the front of St Mark’s Church, Purley – and I was starting to speak at the Memorial Service of my best friend, Chloë.

In front of me were hundreds of faces, young and old alike. They were there to celebrate and remember an ultimately, short but extremely well-lived life.The life of Chloë Drury.

On Wednesday the 27th of February last year, I received a call from a friend, she sat me down, she looked me in the eye and told me that Chloë was going to die, that there was nothing anyone could do anymore and it was going to happen in the next few days.

I sat in shock, I tried to ask questions about when it would happen, and how did they know?

Chloe Drury - Medical Innovation Bill
Chloe Drury – Medical Innovation Bill

All I remember thinking is that I felt desperately helpless. That night I stayed surrounded by friends, trying to find comfort in each other, anything not to be alone.

All night I relived the time I spent with Chloe, the first time we met, the days spent in her company, the last time I ever saw her. I thought about her dreams and aspirations, the things that she’d been so excited about, and which now would never be fulfilled.

When the news finally came through early the next morning that Chloë had passed, there were no more tears, no more uncontrollable emotion just silence, I was completely numb.

Mike Thomas and Liam Ryan at the Houses of Parliament 24th Feb 2014 speaking alongside Lord Saatchi as he launched the public consultation LIVE via Google Hangout.

Over the next few hours I had to have conversations that I wish never had to happen, to be bearer of the worst news possible, to look people in the eye, friends and family alike, and tell them that Chloë had died.

Watching them breakdown in front of me was unbearable.

The following weeks, were the most difficult. As a friendship group we spent nearly every day together, making sure that no one was alone for too long.

Being alone let you think too much, it allowed you to dwell and let reality set in, it let you realise that you would never see Chloë again, that you never got to say goodbye.

These painful thoughts would stay with me for months.

Eventually, we had to return to school, we had to return to everyday life.

But no matter how normal you try to make it, it’s impossible to pretend that nothing has changed.

For me personally, my school attendance was almost non existent, the last place I wanted to be was at school, because at that point in my life, lessons and exams didn’t matter, grades and university places weren’t important, I’d lost a friend and it had stripped me of all motivation. I had no drive.

There were two factors that help me deal with Chloë’s passing. Firstly the unwavering and resolute support that we all received from friends, dealing with this alone would have been undoubtedly impossible.

Secondly, the sheer bravery and optimism of her mum, Debbie Binner.

Despite what she has had to endure, she is always looking for a positive. Debbie has turned loosing Chloë into her driving force.

It was Debbie who urged me to speak in St Mark’s Church, who gave me the opportunity to say goodbye properly. When I stood at that lectern and spoke, I felt like I had finally let Chloë know how much she meant to me.

I hope that I have given you an insight into the pain that losing a friend causes, for young people like me.

And if you remember just one aspect of my words, I urge for it to be this one.

Chloe’s death has brought about many questions about cancer treatments.

And I urge you to ask yourself this question: Have you done everything you could have, were you bold enough, brave enough to demand more, more innovation and more accessibility to cancer treatments, so that more lives are saved where possible?

For Chloë and the hundreds of others who find themselves in her situation, Make sure the answer is yes.

This blog is based on a speech by Mike Thomas, who spoke at the House of Lords on Monday 24 Februrary about the death of his friend Chloë Drury and in support of the Medical Innovation Bill. Chloë died on 28 February 2013, of Ewings sarcoma aged 18. 

Telegraph: Lord Saatchi launches the consultation on his medical innovation bill – live from the House of Lords

Today Lord Saatchi launched his Medical Innovation Bill LIVE via Google Hangout from the House of Lords.

The politician was joined by a panel of experts including Telegraph doctor Max Pemberton in a live video stream from the House of Lords today where they will call on the public to support his Medical Innovation Bill.

Introduced by Lord Saatchi last year following the death of his novelist wife Josephine Hart to ovarian cancer in 2011, the Bill seeks to give medics greater freedom to test out cutting edge treatments on dying patients.

Currently doctors are forced by the threat of legal action to stick to standard procedures, even when they are proving ineffective.

Lord Saatchi’s bill has already received huge public support and health secretary Jeremy Hunt promised to support legislation on the issue after the public consultation which is launched today and ends in May.

Read in the Telegraph 

 

Harrison, Duchenne and I

Harrison and Alex Smith

When Harrison was just 4 years old, I took him to his pediatrician thinking he might have a mild physical delay and may need physical therapy.  Within 2 weeks, a blood test and a visit with a neurologist provided us with the most devastating diagnosis imaginable.  Duchenne muscular dystrophy.  The neurologist explained to us that Harrison’s muscles would rapidly deteriorate, he would lose the ability to walk, to use his arms, to bathe himself, to go to the bathroom on his own. Eventually Duchenne would attack his heart and lungs and the disease would take his life.  We have nothing to stop it he told us.  It’s 100 % fatal, I wish I could tell you it was cancer.

I swung in to action, I challenge myself daily…mentally and physically to do the best I can.  I’ve started a charity, Harrison’s Fund, to generate as much cash as I can to put into breakthrough Duchenne research to find a treatment for this generation of children and young adults. We have already invested hundreds of thousands of pounds. I’ve become a networker, at all times, this never really switches off, I’ve started a not for profit race team, Harrison’s Racing,  I’ve literally gone from  Average Man to Ironman, completing my first Ironman triathlon in October last year. But, becoming just an Ironman doesn’t seem quite enough for me now… next in the challenge list is to take Harrison on an Ironman with me. Drag him in a boat behind me for 3.8km, take him on the 180km bike leg and then push him the no small matter of a 42.2km marathon, all back to back in under 13hrs and 47mins, a world record time. Harrison will also be an Ironman… that’s just cool! I am in the process of assembling the team to get this done, from building a bespoke catamaran to drag, to building the bike and Harrison machine and the running chair needed to take a child that no longer really fits in any of the running chairs out there anymore.

Harrison is now nearly 8, and we live each day with the knowledge that because he’s got a duplication of exon 51 of his dystrophin gene, his symptoms continue to progress and he continues on a steady and rapid physical decline.

Harrison’s doing incredibly well considering but he has reached a plateau all children with Duchenne  do around there seventh and eighth birthdays.

Harrison Smith

This is a stage of progression that is marked by an accelerated decline…  It is marked by …

Shock – the shock of his legs buckling without warning, sending him tumbling to the ground. It is marked by the shock of not being able to open the jar he used to be able to

Fatigue – he is plagued by the fatigue of exerting great effort just to do “normal” things – like keeping his balance and getting up from the floor.

Strategizing – thinking about how we organise and prioritize a day for the family that will not wear him out yet still let him experience all the world has to offer.

Disappointment – the searing realization that he can’t play organized sports, my lack of words or adequate comfort when he walks into the house fighting back tears because his brother or friends just rode off on their bikes and he can’t be with them.

Routine –  daily stretching sessions, and regularly physical therapy appointments and doctor appointments.

Hanging on – to what may be the last time he’ll complete a task or have the energy to help me coach his brother Williams under 6 rugby team.

This is the time when kids are moving faster and pushing limits and growing and looking forward to the future.  And I’m terrified that Harrison may not have one.  Annual events like birthdays and the end of a school year are marked by conflicting emotions – they’re marked by relief that we were given the gift of another year.  And by grieving one less year we have together.

While these symptoms are heartbreaking, I am also filled with the sobering reality that this is MILD compared to what we will face in the very near future.  There is a train racing toward my little boy and I’m running as fast as I can to scoop him up and save him, but I’m acutely aware that I may not make it in time.  We need to affect massive change to help stop the train.

Children at any age – whether they’re 3 or 5 or 7, like Harrison, should be afraid of the dark.  Of the bully on the playground.  Of the monsters under their bed.  But they should NOT be afraid of needles and biopsies and surgeries and falling and breaking bones.  They should NOT be afraid of no longer walking or of being unable to feed themselves or of losing so much strength in their arms that they can no longer hug their mum’s and dad’s.  Most importantly, they should NOT be afraid of dying.

I’m writing this blog today because this medical innovation bill has the very real potential to help doctors and clinicians slow down the train. In our case the risk of doing nothing is not nothing, the risk of doing nothing is fatal. Fatal every single time. You never survive this.

In any innovation in medicine there is an element of risk, and between clinicians and patients there has to be a level of permissible risk particularly when the population is small and the need is great. In terms of acceptance of risk – our community has already demonstrated our philosophy on this – we have one option right now – it causes cataracts and growth stoppage and osteoporosis, and weight gain and immune system suppression – and this drug is still being studied in the Duchenne population. There are debates over dosing and efficacy and this drug doesn’t even change the final outcome for Duchenne patients.  Yet, our children’s doctors prescribe it, and in fact encourage its use.  It’s part of the standard of care for Duchenne.   Through this use of steroids, we’ve already shown that we’re willing to assume risk.  We understand that the long-term risks of steroid therapy are known, and the long-term risks of a new therapy would be unknown.  But it’s also quite straightforward and simple logic that helps us understand that long-term risks can only become clear when something has been used for the long term – and the only way to get to the long term is to begin.  We have to begin somewhere. Sometime.  And the time has to be now.

What we are not willing to do is assume the risk of doing nothing. If a potential therapy shows promise of stabilization or improvement over what would be expected without any treatment, and it shows safety, then patients and parents should be given a choice to try it with long-term studies taking place concurrently.  Because, at the end of every discussion and assessment of a therapy, we must never lose sight of the reality that the risk of having Duchenne far outweighs the risk of most potential treatments.  And our children must be the beneficiaries of our best effort, of our most noble intentions, and of our greatest commitment to safety AND speed. Because at the end of the day, these children are not a statistic.  They are not a commodity. They are not someone’s science experiment.

They could be YOUR boys or grandchildren…..and they may not be, but the responsibility for saving them belongs to all of us.  I believe we are close to a treatment. We are so close that my son Harrison is part of a generation that will either be the last to die from Duchenne or the first to survive.  We must have a great sense of urgency and we must always remember that the children should NOT serve the science, but the science must always serve the children.

With this bill and the innovation it would encourage we have the potential to move forward and reduce Duchenne from a 100% fatal condition to that of a chronic condition. The time is now, we don’t have time to waste, #maketime.

 http://www.harrisonsfund.com

 

Telegraph: End in sight for cervical cancer?

Ian Hampson is finding it hard to contain his excitement. A molecular virologist with a passionate interest in understanding the relationship between viruses and cancer, he thinks that he and his wife, Lynne, may – he stresses the word may – have found a revolutionary self-help therapy for women with pre-cancerous cell changes in the cervix.

Almost by chance, the Hampsons discovered in their lab at St Mary’s Hospital, Manchester, that the drug lopinavir, licensed for the treatment of HIV, attacks the strain of human papillomavirus (HPV) that causes virtually all cases of cervical cancer. An as-yet unpublished clinical trial has found that lopinavir capsules, when inserted into the vagina, appear to kill off abnormal cells in the cervix. There appear to be no side effects.

Cervical cancer is still the main killer of women in the developing world, causing more than 280,000 deaths worldwide annually, so the idea that women might use a simple vaginal pessary to protect themselves against it is an exciting prospect.

But the results of Dr Hampson’s trial are also promising for women in developed countries. Up to 40,000 women in England alone are found to have abnormalities of the cervix every year, following smear tests or biopsies. At present the only treatment for high grade, pre-cancerous changes is invasive – and far from risk-free – surgery.

Sensational news, then, but it has taken Dr Hampson 12 years and a battle against numerous obstacles – including UK red tape, lack of interest and funding – to reach this point. “Worldwide, one woman dies every two minutes of cervical cancer,” he says. “The results of the early trial are staggering, but I might have to wait another six months to get this study published, and another year to get a bigger study off the ground. I want to get this treatment on the road.”

The story begins in 2002, when Lynne, a lecturer in viral oncology at the University of Manchester, was studying HPV16 – the main cancer-causing strain of HPV – and analysing how human proteins interacted with virus proteins.

“It’s well known that viruses like HPV take over human cells by hijacking their ‘waste disposal systems’ to make cells throw away certain proteins inappropriately,” Dr Hampson says. “Human cells use enzymes called proteases to get rid of waste proteins in different ways. Lynne’s results suggested that HPV was enhancing the function of a specific type of protease.

“The findings made me speculate that there might be a drug that could selectively block this particular function of the virus. A class of drugs used to treat HIV, known as protease inhibitors, primarily targets the HIV protease enzyme; but previous work has shown they could also block the same type of human protease, which our results had shown was important for HPV. It was an epiphany.”

At his wife’s suggestion, they got hold of as many protease inhibitors as they could lay their hands on (using a national Aids repository in the US, which supplies small quantities of drugs for research). “We literally smashed up tablets and stuck them into cultures both of HPV and cervical cancer cells,” recalls Dr Hampson. Several seemed to have activity against HPV and cancer, but lopinavir was the most potent. “Lopinavir was remarkably toxic. We could see the cells dying in the lab.”

By 2011, he says, they had shown that lopinavir was “multifunctional”, meaning it inhibited both viral proteins and the proteins in cancer cells. Still nothing had been proven clinically, and as Dr Hampson soon discovered, setting up a patient trial proved difficult.

Lopinavir is made by the US company Abbott Laboratories as part of a combination drug for HIV called Kaletra. The good news was that, as one of the most widely prescribed drugs worldwide and already licensed for both children and adults with HIV, it would not need extensive safety testing. The bad news was that it was only licensed for oral delivery. This formulation would not, at the strength needed, get enough of the drug delivered to the cervix.

“Our work showed we needed a 10-fold higher concentration of the drug at the site of infection – the cervix – than you can get by taking the drug orally,” says Dr Hampson. “The obvious solution was to reformulate the drug, so it could be applied locally as a cream or pessary, and organise a clinical trial.”

But the cost of reformulating the drug meant there was little interest from drug companies, cancer charities or research agencies. Then one of Dr Hampson’s PhD students pointed out that one oral form of the drug available was a soft gelatin capsule, which might “melt very nicely” in the vagina, and could be trialled as a pessary.

There was another obstacle, though. In Europe the makers of the capsule had phased it out in favour of a hard tablet. A generic lopinavir capsule called Lopimune, made by an Indian company, was only licensed for Africa.

“I wanted to bring some Lopimune into the UK for a trial,” says Dr Hampson, “but it would have involved a lot of red tape – and would have cost a fortune. I felt as if I was banging my head against a brick wall.”

The same PhD student, Dr Orora Maranga, pointed out how useful such a self-help treatment – if it worked – would be to women in Africa, where there are few screening programmes to pick up pre-cancerous conditions, or surgical facilities to treat them.

So it was that, near the end of 2011, the Hampsons finally won approval from Kenyatta National Hospital in Nairobi (where Dr Maranga was then a senior registrar) for a preliminary trial of lopinavir in women with pre-cancerous cells in the cervix. In return they would set up a cervical-cancer screening programme – needed to identify women suitable for the trial – and facilities for surgery, if it was needed.

Funding such a trial was difficult, and Dr Hampson is enormously grateful to those who supplied money or equipment. Donors included St Mary’s, Central Lancashire Healthcare Trust, a company called Hologic, a UK philanthropist, Ken Cholerton, and a small charity on the Isle of Wight, the Caring Cancer Trust.

Finally, last year, in a trial overseen by Dr Maranga, more than 800 Kenyan women were screened for cervical cancer. Of those, 21 per cent were found to be HPV positive, of whom 40 had pre-cancerous cells in the cervix. Twenty-three of this group had high-grade disease – where there is a higher risk of cancer developing and for which the only treatment is surgery. A further 17 had low-grade or borderline disease.

All 40 women were asked to insert a lopinavir capsule as a pessary, twice a day, for two weeks. At three months, each participant underwent a biopsy of the cervix, together with a smear test. In 19 of the 23 women with high-grade disease, the abnormal cells had disappeared. Two women now had low-grade changes, while two still had high-grade disease, for which they needed surgery.

Dr Hampson emphasises that this was an early trial, the type normally used to look at how well a new drug is tolerated. And he points out that there were no side effects. “We had a 90 per cent treatment response in the high-grade group – we couldn’t believe it,” he says. Although high-grade disease can sometimes disappear spontaneously after a year or two, this is rare after three months. There was also, he reports, “very high clearance” among women with low-grade and borderline cell changes.

Dr Hampson is now planning a larger controlled trial involving 500-1,000 women based in three sites in Africa – Nairobi, Kampala and Johannesburg. If he can raise the funds, that is. If the results can be replicated, the advantages for women everywhere are obvious. This potential treatment, less invasive than surgery, would also avoid the risk of premature labour in pregnancy that is associated with the operation.

“Research has found that one surgical excision of abnormal cervical cells increases the risk of premature delivery by 14 per cent,” says Prof Pierre Martin-Hirsch, a gynaecology oncologist at Lancashire Teaching Hospitals, who finds the results of this first trial “impressive”.

By contrast, Dr Hampson points out that in cases where the disease is persistent, “treatment with lopinavir can be repeated as often as needed, because it appears to be safe.”

Lopinavir could also be an option for women with low-grade pre-cancers who are currently offered a “watch and wait” approach – “a horrible time of anxiety”.

“While a vaccine is now being offered against HPV in some parts of the world,” he points out, “it will take 20 to 30 years before we have immunity.”

Cancer Research UK says it cannot comment on a trial that has not yet been peer-reviewed or published. “This is raising the hopes of patients,” warns a spokesman. “A peer review might find flaws in the trial. It may never get published.”

What frustrates Dr Hampson is the fact that, even if further trials were successful, it could be years before this pessary becomes available. There is now talk of of a “window trial” – one in which lopinavir could be offered to women with high-grade disease while they are waiting for surgery. “The drug has been shown to be safe, and the women have nothing to lose and everything to gain. If it works they will not need an operation.

“We are looking at this possibility, but clinicians in the UK are understandably cautious about using treatments off-label. And who can blame them? In our litigation culture, it doesn’t pay to take risks.”

Which is why Dr Hampson supports Lord Saatchi’s Medical Innovation Bill, designed to encourage doctors to innovate without fear of prosecution, and which is the subject of a government-backed consultation.

“At present there is a great reluctance to deviate from the accepted best clinical practice. Had we been able to obtain the particular drug formulation in the UK, it would have been far easier to convince a clinician to try the treatment – if the fear of litigation was reduced.”

The University of Manchester has filed a patent for treatment with lopinavir for HPV-related disease

Saatchi’s Medical Innovation Bill

Lord Saatchi’s Bill, now open to public consultation, would allow doctors to prescribe ‘’off label’’ drugs. These are drugs that have been tried and tested for one specific use (in this case lopinavir used to combat HIV). lopinavir is known to be safe, but doctors cannot easily prescribe it for other, non-standard uses. The Bill aims to speed up the development of new and innovative treatments for cancer and other diseases.

The Caring Cancer Trust has agreed to accept donations towards funding a phase 3 trial of lopinavir for pre-cancerous cell changes in the cervix. More details are available from their Virgin Money Giving page which can be accessed from their website http://www.caringcancertrust.com/

Guest blog: Fearful doctors ‘in a war against inertia’

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By Dr Lefever

The Medical Innovation Bill, to be introduced by Lord Saatchi, is intended to help patients by allowing their doctors to be less constrained by clinical orthodoxy.

Single issue fanatics, whose fervent beliefs have no scientific merit, will still be reined in. But doctors who can make a reasonable case for a non-standard approach will be able to do so. Clearly they have to have the support of the patient. Also they must convince a scrutinising panel

The Medical Innovation Bill intends to make it easier to define what is sensible and permissible innovation and, by contrast, what is reckless experimentation.

It would do this by setting out a clear protocol to follow before offering the patient an innovative or experimental procedure.

Under the bill, the lead clinician must consult with, and gain approval from, a multi-disciplinary team. Without approval, the innovation cannot be legally offered.

This approach is eminently sensible. Which of us would not want to brush aside ‘the way things have always been done’ if doing so might bring additional years of health and happiness to a loved one?

Doctors in the front line, right alongside their patients in the battle with disease and decay, want to do the best they can. So who is the enemy? Who has a vested interest in the status quo? Whose head would roll if something did not work out as hoped when something new is tried?

The answer at present is that we are witnessing a civil war. The doctor’s own career would be sacrificed if he or she steps out of line.

The National Institute for Health and Clinical Excellence (NICE) was intended to weed out mavericks. It has become the keeper of the purse for the NHS.

The General Medical Council has responsibility for putting doctors names onto the medical register, through monitoring medical education, as well as for striking off miscreants. Yet how much attention do they pay to considering whether doctors are properly trained for the job they actually do? For example, are GPs trained to counsel? Hardly at all. Yet that is half the work. Are specialists trained to take risks? No. But they have to.

Lawyers and Coroners judge the actions of doctors on what their colleagues would customarily do.

A profound inertia results from the limited vision of these guardians of public safety – with the consequence, as Lord Saatchi understands only too well, that doctors now fear more for themselves and their families than they do for their patients.

About the author

Dr Robert Lefever is regarded as the pioneer of addiction treatment methods and rehab centres in the UK. He established the very first rehabilitation centre that treated patients with eating disorders alongside those with drug and alcohol problems. He was also the first to treat compulsive gambling, and workaholism.

READ: Dr Lefever blogs for the Daily Mail

READ: Dr Lefever’s own blog

READ: http://www.doctor-robert.com/